The present disclosure provides improved genome editing compositions and methods for editing a PCSK9 gene. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of hypercholesterolemia or a condition associated therewith.

The present invention relates to genetically modified ascomycetous filamentous fungi, particularly of the species Thermothelomyces heterothallica, having reduced activity or expression of KEX2 and/or ALP7, said filamentous fungi is capable of producing elevated amounts and stability of an exogenous protein.

The current invention relates to methods and compositions for converting plant derived biomass to textile grade fibres by an enzymatic single bath method, which does not require draining, refilling or water washes after every treatment. The single bath conversion of raw natural fibres derived from plant based biomass to softer, finer textile grade fibres is an eco-friendly process which is efficient and conserves water. The textile grade fibres produced have same quality when compared to those derived from conventional multi bath process.

Provided herein are compositions for gene modification or editing and methods of using same to treat or prevent certain conditions. Specific compositions and methods capable of safely and effectively editing gene targets expressed in the liver to durably lower LDL-C thereby treating a leading cause of cardiovascular disease are disclosed.

Disclosed are methods and isolated cells useful for the improved production of function fXa derivative protein that acts as a fXa inhibitor antidote. One aspect relates to an isolated cell comprising the r-Antidote polynucleotide and Furin polynucleotide. Another aspect relates to a method for preparing the cleaved two-chain r-Antidote by expressing, in a cell, the pre-processed r-Antidote polypeptide and a Furin polypeptide.

A targeted delivery of recombinant therapeutic protein to inhibit the growth of cancer cells is disclosed. The recombinant protein derived from Abrin-a A-chain able to inhibit the growth of cancer cell and conjugated to stxB to form Abrin-f-stxB. The abrin toxin A chain and the Shiga toxin B chain are linked with the furin linker to form a recombinant therapeutic protein as chemotherapeutic agent to inhibit the growth of cancer cells. A conjugate for the delivery of a compound into cancer cells, comprising a first module mediates cell targeting and facilitates cellular uptake, a second module facilitates transport to the endoplasmic reticulum (ER), a third module mediates translocation from the ER to the cytosol and a compound that is desired to be delivered to the cytosol. The recombinant protein Abrin-f-stxB exhibits high binding affinity of stxB as a drug delivery system to Gb3 receptor in targeted cancer diagnosis and treatment.

Disclosed are methods and isolated cells useful for the improved production of function fXa derivative protein that acts as a fXa inhibitor antidote. One aspect relates to an isolated cell comprising the r-Antidote polynucleotide and Furin polynucleotide. Another aspect relates to a method for preparing the cleaved two-chain r-Antidote by expressing, in a cell, the pre-processed r-Antidote polypeptide and a Furin polypeptide.

The invention belongs to the biopharmaceutical. It involves the role and mechanism of PCSK9 in inflammatory immune diseases, and the application of PCSK9 inhibitors to the preparation of drugs for the treatment of inflammatory immune diseases mediated by T cells. In particular, it involves the use of PCSK9 monoclonal antibody, PCSK9 interference RNA and PCSK9 small molecule inhibitors for treating psoriasis, atopic dermatitis, or urticaria. The invention uses psoriasis as an embodiment for the study of inflammatory and immune diseases. It is found that PCSK9 plays an important role in the treatment of inflammatory immune diseases. The PCSK9 monoclonal antibody, PCSK9 interference RNA, and PCSK9 small molecule inhibitor can be further developed for treating inflammatory immune-diseases, such as psoriasis with fewer adverse reactions, at low cost, and with good efficacy.

The present invention provides multispecific antigen-binding molecules and uses thereof. The multispecific antigen-binding molecules comprise a first antigen-binding domain that specifically binds a target molecule, and a second antigen-binding domain that specifically binds an internalizing effector protein. The multispecific antigen-binding molecules of the present invention can, in some embodiments, be bispecific antibodies that are capable of binding both a target molecule and an internalizing effector protein. In certain embodiments of the invention, the simultaneous binding of the target molecule and the internalizing effector protein by the multispecific antigen-binding molecule of the present invention results in the attenuation of the activity of the target molecule to a greater extent than the binding of the target molecule alone. In other embodiments of the invention, the target molecule is a tumor associated antigen, and the simultaneous binding of the tumor associated antigen and the internalizing effector protein by the multispecific antigen-binding molecule of the present invention causes or facilitates the targeted killing of tumor cells.

Materials and methods of in vivo possessing of target proteins in plants by co-expressing with proprotein processing enzyme, human Furin, are provided. A method of expressing highly soluble and functional active human Furin in plants also is provided.